Voretigene neparvovec - Spark Therapeutics

Drug Profile

Voretigene neparvovec - Spark Therapeutics

Alternative Names: AAV2 hRPE65v2; Leber congenital amaurosis gene therapy; LUXTURNA; SPK-RPE65

Latest Information Update: 20 Nov 2017

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At a glance

  • Originator The Childrens Hospital of Philadelphia
  • Developer Spark Therapeutics
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Leber congenital amaurosis; Retinitis pigmentosa
  • New Molecular Entity No

Highest Development Phases

  • Preregistration Leber congenital amaurosis

Most Recent Events

  • 10 Nov 2017 Updated efficacy and adverse events data from a phase III trial in Leber congenital amaurosis released by Spark Therapeutics
  • 07 Nov 2017 EMA validates the MAA for voretigene neparvovec for Leber congenital amaurosis for review
  • 12 Oct 2017 US FDA's Cellular, Tissue and Gene Therapies Advisory Committee recommends approval of voretigene neparvovec for the treatment of patients with Leber congenital amaurosis
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